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41 results
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Orphan designation: Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene for: Treatment of beta thalassaemia intermedia and major
Date of first decision: 24/01/2013, Positive, Last updated: 03/06/2019 -
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Orphan designation: Eculizumab for: Treatment of atypical haemolytic uraemic syndrome (aHUS)
Date of first decision: 24/07/2009, Positive, Last updated: 20/11/2019 -
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Orphan designation: Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] (ponatinib) for: Treatment of chronic myeloid leukaemia
Date of first decision: 02/02/2010, Positive, Last updated: 23/03/2015 -
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Orphan designation: (1R,2S) 6-bromo-alpha-[2-(dimethylamino)ethyl]-2-methoxy-alpha-(1-naphthyl)-beta-phenyl-3-quinolineethanol bedaquiline for: Treatment of tuberculosis
Date of first decision: 26/08/2005, Positive, Last updated: 01/04/2014 -
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Orphan designation: carfilzomib for: Treatment of multiple myeloma
Date of first decision: 03/06/2008, Positive, Last updated: 02/12/2015 -
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Orphan designation: Phosphorothioate oligonucleotide targeted to transthyretin (inotersen) for: Treatment of ATTR amyloidosis
Date of first decision: 26/03/2014, Positive, Last updated: 17/05/2019 -
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Orphan designation: Recombinant IgG degrading enzyme of Streptococcus pyogenes (imlifidase) for: Prevention of graft rejection following solid organ transplantation
Date of first decision: 12/01/2017, Positive, Last updated: 28/08/2020 -
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Orphan designation: budesonide for: Treatment of eosinophilic oesophagitis
Date of first decision: 05/08/2013, Positive, Last updated: 18/01/2018 -
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Orphan designation: Cyclopropane-1,1-dicarboxylic acid [4-(6,7-dimethoxy-quinolin-4-yloxy)-phenyl]-amide (4-fluoro-phenyl)-amide, (L)-malate salt (cabozantinib) for: Treatment of medullary thyroid carcinoma
Date of first decision: 06/02/2009, Positive, Last updated: 12/05/2014 -
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Orphan designation: Eculizumab for: Treatment of neuromyelitis optica spectrum disorders
Date of first decision: 05/08/2013, Positive, Last updated: 20/11/2019 -
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Orphan designation: Chimeric anti-interleukin-6 monoclonal antibody (siltuximab) for: Treatment of Castleman's disease
Date of first decision: 30/11/2007, Positive, Last updated: 11/07/2014 -
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Orphan designation: Recombinant human tripeptidyl-peptidase 1 for: Treatment of neuronal ceroid lipofuscinosis type 2
Date of first decision: 12/03/2013, Positive, Last updated: 09/02/2016 -
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Orphan designation: Ketoconazole for: Treatment of Cushing's syndrome
Date of first decision: 23/04/2012, Positive, Last updated: 22/09/2020 -
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Orphan designation: Eculizumab for: Treatment myasthenia gravis
Date of first decision: 29/07/2014, Positive, Last updated: 20/11/2019 -
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Orphan designation: Antisense oligonucleotide targeted to the SMN2 gene (nusinersen) for: Treatment of 5q spinal muscular atrophy
Date of first decision: 02/04/2012, Positive, Last updated: 16/10/2018 -
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Orphan designation: velaglucerase alfa for: Treatment of Gaucher disease
Date of first decision: 06/06/2010, Positive, Last updated: 23/06/2010 -
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Orphan designation: Recombinant human nerve growth factor (cenegermin) for: Treatment of neurotrophic keratitis
Date of first decision: 14/12/2015, Positive, Last updated: 02/02/2016 -
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Orphan designation: Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] (ponatinib) for: Treatment of acute lymphoblastic leukaemia
Date of first decision: 02/02/2010, Positive, Last updated: 23/03/2015 -
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Orphan designation: Recombinant human parathyroid hormone for: Treatment of hypoparathyroidism
Date of first decision: 16/01/2014, Positive, Last updated: 26/04/2017 -
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Orphan designation: 3-[5-(2-Fluoro-phenyl)-[1,2,4]oxadiazole-3-yl]-benzoic acid (ataluren) for: Treatment of Duchenne muscular dystrophy
Date of first decision: 27/05/2005, Positive, Last updated: 10/03/2015 -
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Orphan designation: ciclosporin for: Treatment of vernal keratoconjunctivitis
Date of first decision: 06/04/2006, Positive, Last updated: 12/07/2018 -
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Orphan designation: Phosphorothioate oligonucleotide targeted to apolipoprotein C-III (volanesorsen) for: Treatment of familial chylomicronaemia syndrome
Date of first decision: 19/02/2014, Positive, Last updated: 20/05/2019 -
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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA (also known as patisiran) for: Treatment of transthyretin-mediated amyloidosis
Date of first decision: 06/04/2017, Positive, Last updated: 30/05/2018 -
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Orphan designation: Humanised monoclonal antibody against P-selectin (crizanlizumab) for: Treatment of sickle cell disease
Date of first decision: 09/08/2012, Positive, Last updated: 24/11/2020 -
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Orphan designation: (S)-2-nitro-6-(4-trifluoromethoxy)benzyloxy)-6,7-dihydro-5H-imidazo[2,1-b] [1,3] oxazine (pretomanid) for: Treatment of tuberculosis
Date of first decision: 29/11/2007, Positive, Last updated: 09/12/2020